Five years ago, Hemophilia Federation of America articulated a broad vision that includes advocating for improved care and quality of life for those affected by bleeding disorders. Hemophilia imposes many burdens: painful and unpredictable bleeding; joint damage or other serious long-term complications; onerous treatment regimens; and numerous other constraints on personal and family life. HFA shares our community’s eager hope for a safe, effective, single-dose therapy that could relieve these burdens and allow affected individuals to thrive.
Yesterday, the U.S. Food and Drug Administration approved HEMGENIX, the first gene therapy for hemophilia B. This marks a momentous step toward realizing the hopes of many. HFA welcomes the introduction of this innovative treatment option.
At the same time, HFA also recognizes that substantial uncertainties persist with respect to this potentially transformative new therapy.
We know that not all individuals with hemophilia B will be eligible for the new therapy. We know that some people, even if eligible, will decide that the new therapy does not meet their personal treatment goals or timelines. Other individuals may opt to wait until more is known about the gene therapy’s real-world efficacy and/or long-term safety record. In all cases, the decision whether or not to opt for gene therapy must rest with the patient and their health care provider.
As HFA marks this potentially promising new chapter in hemophilia treatment, we remain always mindful of our community’s history. We urge all who are involved with gene therapy – regulators, drug sponsors, insurers, and health care providers – to center their actions on patient well-being. HFA will continue to engage with these diverse stakeholders to ensure that patient health, safety, and interests always come first.